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The HSE said the current price of Spinraza – more than €20 million over five years – is not cost-effective.

FAMILIES OF CHILDREN with a rare genetic condition have criticised the HSE’s decision not to reimburse a drug that could improve their quality of life.

About 26 children in Ireland have Spinal Muscular Atrophy (SMA) – a life-threatening and debilitating disease that causes progressive muscle weakness and loss of movement due to muscle wasting – and could benefit from Spinraza (also known as Nusinersen).

The medication is an orphan drug, meaning it is commercially underdeveloped and therefore very expensive. About one in 11,000 babies are affected by SMA. It is the number one genetic cause of death of infants and is in the same family as motor neurone disease.

More than 20 countries in Europe have reimbursed Spinraza. Access to the medication has been approved in all but 3 EU countries – Ireland, Estonia and the UK (bar Scotland). Two young girls in Ireland with were granted access to the drug in January 2017 as part of an emergency programme.

The HSE said it does not deem the drug to be cost-effective, as it would cost over €20 million over five years. Parents, who have been campaigning for access to the drug for years, said they are very disappointed by the decision.

SMA Ireland is planning to demonstrate outside Leinster House at 11am on Thursday, which is World Rare Disease Day. Members will present a petition calling on the government to grant access to Spinraza; it has almost 90,000 signatures to date.

The most common form of the disease is 5q SMA, which has four different types: 1, 2, 3 and 4– based on age of onset and the highest physical milestone achieved. Type 1 is the most severe form of SMA and accounts for between 50–70% of cases of childhood onset SMA. About 95% of children with Type 1 who are untreated die before the age of two.

Source: SMA Ireland

Spinraza is delivered through the lower back via a lumbar puncture. Children who have been granted access to the medication have generally responded well, with some gaining strength and motors skills and reaching milestones previously thought impossible.

Cost and clinical trials

In a statement the HSE said it “regrets” that it has to date been unable to come to an agreement with the drug’s manufacturer, Biogen. The statement notes “the limitations of the current evidence on clinical effectiveness, and the high price being charged by the pharmaceutical company”.

The HSE said when assessing Spinraza, the organisation’s Drugs Group focused on two areas: reviewing the evidence of the clinical effectiveness of drug (the benefits for patients undergoing clinical trials) and the cost-effectiveness of the drug.

The HSE said the medication would cost €600,000 per patient for the first year and €380,000 per patient per year thereafter, and so would have an estimated budget impact in excess of €20 million over a five-year period.

A spokesperson said, in comparison, the HSE’s total expenditure for complex paediatric homecare packages in 2018 was €23.8 million.

While the HSE is anxious to provide all possible support to this very vulnerable group of patients and those who care for them, the decision reached by the Leadership Team concurs with the recommendation of the Drugs Group, which is that reimbursement could not be approved at the price currently being charged by the pharmaceutical company.

The spokesperson added that the HSE wrote to Biogen last Thursday informing it that the medication would not be reimbursed at the current price.

Under the requirements of the Health (Pricing and Supply of Medical Goods) Act 2013, the company has 28 days to respond or make representations to the HSE’s proposed decision.

‘Figures out of date’

Biogen said it is “genuinely surprised” by the HSE’s statement. “The figures referenced are at least six months out of date. In terms of efficacy, the effectiveness of nusinersen in type II/III SMA patients is supported by a randomised clinical trial in the same way that efficacy has been demonstrated in type I SMA patients,” a statement notes.

It adds that both trials were stopped early on the advice of the US Food and Drug Administration and the European Medicines Agency “given the strength of the interim results”.

The statement also notes that Spinraza has been approved for reimbursement in 25 other European countries, saying the price being paid in those countries is in line with what was offered to the HSE after negotiations.

Biogen recognises that this decision marks a deeply concerning day for SMA patients and their families, and we share that concern: Ireland is now significantly behind many parts of the world in making nusinersen available.

Biogen asked the HSE to re-enter discussions “as a matter of utmost priority”.

Fiona Bailey, whose son Sam has SMA Type 2, this evening called on the HSE to reverse its decision urgently, noting the “debilitating and degenerative” nature of the condition.

“Our son Sam, who’s [ninth] birthday is next week, needs Spinraza now, it is the only drug to treat Spinal Muscular Atrophy,” she said.

Fiona previously told TheJournal.ie she understands that Spinraza is expensive but doesn’t believe the price should be the main focus of the discussion.

It’s a lot of money, but if a child has cancer would someone say they can’t have chemotherapy because it’s too dear? Of course not.

“We’re basically being told they can’t have the medication they need, full stop. I understand it’s dear but, as a doctor said to me, a night in ICU is dear, a week in ICU is dear, two weeks in ICU is dear.

“We can expect at least two more stints in ICU in 2019, if we’re lucky. Sam will get to come with us again, if we’re lucky.”

Cost of National Children’s Hospital

Elaine McDonnell, CEO of Muscular Dystrophy Ireland, said it is “unconscionable that new therapies which we know would make a huge difference to the lives of people with SMA are being denied to them”.

And as one of the last countries to hold out on approval for these therapies, it is clear that Ireland is now at odds with regulators, health authorities and experts across Europe.

“Our children deserve to be treated no less than their fellow citizens in other European countries and deserve the health benefits and life experiences that can be afforded by Spinraza, that otherwise may not be attainable.

“There is a tragic irony here in that our government and the HSE have found every penny possible to build a National Children’s Hospital, but are denying the funds necessary to deliver the treatments to the patients for whom the hospital is being built,” McDonnell said, calling on Health Minister Simon Harris to intervene.

‘A price has now been put on the value of life’

Sinn Féin TD Caoimhghín Ó Caoláin is among the TDs to raise Spinraza in the Dáil. Speaking today, he said parents’ hopes “have now been dashed after months of relentless campaigning”.

A price has now been put on the value of life and wellbeing. This decision is morally wrong and has again trapped very ill patients in the middle of a bidding war between Government and the pharmaceutical industry.

Fianna Fáil TD John Brassil also criticised the decision and urged the government to ensure that a Bill which would improve the availability of orphan drugs for rare diseases in Ireland “progresses through the Oireachtas without delay”.

“Spinraza has been one of the more high-profile drugs which has caught the public’s attention, mainly because the families of children suffering with rare neuromuscular disorders have been campaigning so effectively.

“But they should not have to do that. They, and many others, suffering from rare conditions should be able to access the medication they need,” Brassil said.

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